• 11:30 AMTrial architecture for small, heterogeneous populations — lessons from NPC, sphingolipidoses, and rare neurodegenerative LSDsRaphael Schiffmann, MD · Texas Christian University, USA
• 11:50 AMAdaptive, pragmatic, and decision-focused trial designsTerrence Eagleton · Medpace, Ohio, USA
• 12:10 PMUse of composite endpoints in Gaucher disease and other rare disordersAri Zimran, MD · Shaare Zedek Medical Center, Israel
• 12:30 PMSession Q&A — Open discussion with all session speakers

Measuring meaningful benefit beyond biochemical correction

• 1:30 PMAppropriate use of natural history data as external controls: when it works and when it failsDavid Kronn, MD · New York Medical College, USA
• 1:50 PMEndpoint selection in pediatric-onset lysosomal disorders and prediction of adult outcomesUma Ramaswami, MD · Royal Free London NHS Foundation Trust, UK
• 2:10 PMPatient-centered outcomes and trial burden in lysosomal disordersTanya Collin-Histed · International GARDIAN, UK
• 2:30 PMSession Q&A — Open discussion with all session speakers

Biomarkers as decision-support tools; distinguishing disease progression from treatment-related risk

• 3:15 PMPharmacodynamic vs. disease-burden biomarkers: Fabry disease as the model caseBehzad Najafian, MD · University of Washington, USA
• 3:35 PMRenal biomarker harmonization and longitudinal interpretationDavid Warnock, MD · University of Alabama, USA
• 3:55 PMBiomarkers for patient selection and enrichment in NPC trials: oxysterols, NfL, and cholestane-triolCaroline Hastings, MD · UCSF Benioff Children’s Hospital, California, USA
• 4:15 PMInterpreting biomarkers in treated and late-treated patients: when numbers misleadNeal Joseph Weinreb, MD · University of Miami, USA
• 4:35 PMSession Q&A — Open discussion with all session speakers

Partial responders, CNS vs. peripheral benefit, and durability

• 9:00 AMThe clinical trial endpoint journey, and lessons learned on how to design a trial for approval for small moleculesDominique P. Germain, MD, PhD · APHP - University Paris-Saclay, France
• 9:20 AMLessons from GBA1-associated Parkinson disease trialsAndres Felipe Deik, MD · University of Pennsylvania, USA
• 9:40 AMNPC as the case study: NALL, the NPCCSS endpoint journey, and lessons from a CRL to first FDA approvalFrances Platt, PhD · University of Oxford, UK

Immunogenicity, residual disease, and real-world effectiveness

• 10:00 AMResidual disease in treated Fabry patients and implications for future trialsProfessor Derralynn Hughes · Royal Free Hospital and University College, UK
• 10:20 AMNeuronopathic lysosomal disorders in the treated era: biomarkers, neurocognition, and residual diseaseJoseph Muenzer, MD, PhD · University of North Carolina at Chapel Hill, USA
• 10:40 AMChallenges in global clinical trial execution for sphingolipidosesFatih Ezgü, MD · Gazi University Faculty of Medicine, Turkey
• 11:00 AMSession Q&A — Open discussion with all session speakers

Clinical management after dosing

• 11:45 AMDurability of response and loss of efficacy following gene therapyArian G Pano, MD · Georgep Consulting LLC, Massachusetts, USA
• 12:05 PMWhen stabilization equals success: endpoints in neurodegenerative lysosomal disordersRoberto Giugliani, MD · Serv Genet Med HCPA / Dep Genet UFRGS, Brazil
• 12:25 PMPost-trial monitoring and real-world implementation following gene therapyTahsin Muzaffar, MD · Industry Advanced Therapy Programs
• 12:45 PMImmune risk stratification and patient eligibility criteria for gene therapy trialsCoy D. Heldermon, MD, PhD · University of Florida, USA
• 1:05 PMSession Q&A — Open discussion with all session speakers
• 1:25 PMClosing Remarks & Adjournment — Program Chair